Jazz Pharmaceuticals to Acquire Actio Biosciences for Up to $1.32 Billion in Rare Epilepsy Deal

Jazz Pharmaceuticals plc has agreed to acquire Actio Biosciences, Inc. for $820 million upfront and up to $500 million in milestones. The deal, expected to close in late 2026, centers on ABS-1230, a clinical-stage KCNT1 inhibitor for KCNT1+ epilepsy, an ultra-rare genetic disease affecting 2,500 U.S. patients with no approved therapies. Actio will spin out its remaining assets, including a Charcot-Marie-Tooth candidate, into a new private entity in which Jazz will hold a minority stake.

Actio Biosciences acquisition terms

• ▪The transaction between Jazz Pharmaceuticals plc and Actio Biosciences, Inc. was unanimously approved by both boards and is expected to close in the fourth quarter of 2026.
• ▪Jazz Pharmaceuticals plc plans to fund the acquisition of Actio Biosciences, Inc. using cash on hand and existing financing facilities.
• ▪The acquisition agreement between Jazz Pharmaceuticals plc and Actio Biosciences, Inc. includes up to $500 million in contingent approval and sales milestone payments.
• ▪Jazz Pharmaceuticals plc entered into a definitive agreement to acquire privately held Actio Biosciences, Inc. for $820 million upfront in cash.

ABS-1230 clinical development

• ▪ABS-1230 demonstrated seizure reductions in an early clinical proof-of-concept trial in children with KCNT1 epilepsy.
• ▪The ongoing Phase 1b/2a KYRON trial of ABS-1230 is designed to serve as the registrational study to support a U.S. new drug application.
• ▪Actio Biosciences, Inc.'s lead clinical asset, ABS-1230, is a first-in-class small molecule KCNT1 ion channel inhibitor.
• ▪ABS-1230 has secured FDA Fast Track, Rare Pediatric Disease, and Orphan Drug Product designations, and is accepted into the FDA's Rare Disease Evidence Principles program.

KCNT1+ epilepsy disease characteristics

• ▪There are currently no U.S. Food and Drug Administration approved therapies for KCNT1+ epilepsy.
• ▪KCNT1+ epilepsy is a rare genetic developmental and epileptic encephalopathy affecting approximately 2,500 patients in the United States.
• ▪Approximately 80% of individuals with KCNT1+ epilepsy experience disease onset during infancy, often experiencing dozens to hundreds of daily seizures resistant to existing medications.

Jazz rare epilepsy portfolio strategy

• ▪Epidiolex, acquired by Jazz Pharmaceuticals plc in its 2021 buyout of GW Pharmaceuticals, generated $292 million in the second quarter of 2026.
• ▪The acquisition of ABS-1230 expands Jazz Pharmaceuticals plc's rare epilepsy portfolio, building upon its existing epilepsy treatment Epidiolex.

Actio spinout structure

• ▪The new spun-out entity will focus on genetic rare neurological diseases, including the clinical-stage TRPV4 inhibitor ABS-0871 for Charcot-Marie-Tooth disease type 2C.
• ▪At closing, Actio Biosciences, Inc. will spin out a new privately held company retaining certain management, employees, and assets, excluding ABS-1230.
• ▪Jazz Pharmaceuticals plc will receive a minority stake and certain related rights in the new spun-out private company.